FamiliesResearch Clinical trial update from Takeda Since the announcement that the phase 2 trial with TAK-611 for children with Metachromatic Leukodystrophy…MLD adminMarch 20, 2024
FamiliesResearch A Rare Find: The Newborn Screening Collaborative comedy short The UK currently screens newborns for just nine rare but serious conditions whereas the USA…MLD adminDecember 4, 2023
FamiliesResearch A Rare Find: Comedy short wants to create conversation around newborn screening The UK currently screens newborns for just 9 rare but serious conditions whereas the USA…MLD adminNovember 22, 2023
Research Genomics England includes MLD on its provisional list The Generation Study, a project by Genomics England, will see the genomes of 100,000 newborns…MLD adminOctober 4, 2023
Research Gene Therapy Study for Late Juvenile Metachromatic Leukodystrophy (LJ MLD) Following the wonderful news that Gene Therapy was approved for use in England by the…MLD adminFebruary 22, 2022
Research NICE final guidance approves life-changing gene therapy for treating Metachromatic Leukodystrophy (MLD) We are delighted to announce that Atidarsagene autotemcel (Libmeldy®) has been recommended as an option…MLD adminFebruary 4, 2022
Research Newborn Screening for MLD Update A formal application to add Metachromatic Leukodystrophy to the UK Newborn Bloodspot (NBS) programme has…MLD adminDecember 9, 2021
Research Update on approval of gene therapy for MLD NICE (National Institute of Clinical Excellence) the drug decision making body have publicly released their…MLD adminJuly 14, 2021
FamiliesResearch MLD UK registry news Following long discussions around the current worldwide status of Registries for MLD and because of…MLD adminJuly 12, 2021
FamiliesResearch Treating early onset MLD We are pleased to report Orchard Therapeutics has received EC full (standard) market approval for…MLD adminDecember 21, 2020